Comparison of Weekly Somatrogon to Daily Genotropin in Children Born Small for Gestational Age or With Idiopathic Short Stature. (MISSION)

Clalit Health Services

Status and phase: Not yet enrolling; Phase 3

Conditions: ISS; SGA

Treatments: Drug: Somatrogon; Drug: Genotropin

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT07226089

0081-24-RMC

Details and patient eligibility

About

This study is a randomized, open-label, active controlled, parallel group study comparing the efficacy and safety of once weekly Somatrogon to daily Growth Hormone (Genotropin) in pre-pubertal children with short stature either born Small for Gestational Age (SGA) or with Idiopathic Short Stature (ISS). The planned study duration is 12 months with a screening period of up to 30 days. The study will consist of two groups: 140 children with SGA who are naïve to GH treatment will be randomized 1:1 to receive either Somatrogon or Genotropin for 12 months. A second group will include 114 children with ISS who are naïve to GH treatment who will be randomized 1:1 to receive either Somatrogon or Genotropin for 12 months.

Enrollment: 254 estimated patients

Sex: All

Ages: 3 to 11 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Diagnosis of SGA or ISS. SGA, defined as born with a birth weight and/or length <-2 SDS below the mean for gestational age. ISS, defined as height < -2 SDS for age and gender without evidence of GHD
2. Females aged ≥3 years and <9 years. Males aged ≥3 years and <11 years
3. Pre-pubertal- Tanner stage 1 for breasts and testes.
4. A bone age of not more than chronological age recorded in previous 8 weeks.
5. Current height < -2 SDS for age and gender.
6. Participants using hormonal replacement therapy(s) must be on an optimized and stable treatment regimen (hormone levels within normal ranges on screening) for at least three months prior to screening
7. Participants who are willing and able to comply with all scheduled visits, treatment plan, laboratory tests, and other study procedures.

Exclusion criteria

1. History of cancer, radiation therapy or chemotherapy.
2. History of GHD.
3. Children who are small due to malnutrition, defined as a Z score of weight for height and/or BMI below -2 for age, according to national standards.
4. History of HIV-positive, acquired immune deficiency syndrome (AIDS), hepatitis B, hepatitis C, or tuberculosis.
5. Microcephaly (Head Circumference < -2 SDS)
6. Any chronic disease or diagnosis, likely to affect growth, including but not limited to gastrointestinal disorder, celiac disease, untreated thyroid disease, diabetes mellitus and metabolic disorders.
7. Known or suspected skeletal dysplasias
8. Known or suspected chromosomal abnormalities
9. IGF-1 >2 SDS
10. Any disorder or condition which, in the opinion of the investigator, might jeopardize participant's safety or compliance with the protocol
11. Prior exposure to growth promoting therapy
12. Current use of any prohibited concomitant medication(s): Any rhGH or growth-promoting therapy, Any therapy that affects appetite or weight, Psychiatric medications associated with weight changes and/or diabetes, excluding medications used to treat ADHD, Any androgen or estrogen therapy including over the counter supplements, Systemic corticosteroids (inhaled or oral) exceeding the doses: Inhaled: > 400 μg/day of inhaled budesonide or equivalent. Oral: > 8 mg/m2/day of oral hydrocortisone or equivalent.
13. Previous administration with an investigational drug within 90 days.
14. Fasting blood glucose >126 mg/dL
15. Renal impairment
16. Hepatic dysfunction.
17. Pregnancy
18. Known hypersensitivity to the components of the study intervention

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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254 participants in 2 patient groups

Somatrogon

Experimental group

Description:

Somatrogon will be administered subcutaneously (s.c) once weekly using a multi dose disposable prefilled pen for single patient use intended for SC self injection.

Treatment:

Drug: Somatrogon

Genotropin

Active Comparator group

Description:

Genotropin will be administrated subcutaneously (s.c) daily using Genotropin Pen growth hormone delivery devices or Genotropin two chamber cartridges (will be supplied in their primary commercial packaging)

Treatment:

Drug: Genotropin